HSESID: Database for HLH Immunodeficiency Disease

A Registry for Hemophagocytic Lymphohistiocytosis (HLH)

Categories:

Immunodeficiency Diseases

Genetics & Genomics

Eligibility Overview:

  • Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis
  • Meets at least 4 of 8 diagnostic criteria for HLH as defined by the Histiocyte Society and is receiving HLH-directed therapy
  • High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy
  1. Brief Summary

    This study is an international effort to collect clinical information on HLH patients. There will be two documentation time-points: the first will follow initial treatment and the second after one year. That data will be analyzed to determine the feasibility of a future randomized clinical trial to evaluate current HLH standard of care.

    Primary Objective

    • To collect data relevant for the assessment of feasibility and design of future interventional studies on HLH treatment

    Eligibility Criteria

    Inclusion criteria include:

    • Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis (HLH)
    • Meets at least 4 of the 8 diagnostic criteria for HLH and is receiving HLH-directed therapy (Steroids are sufficient.)
    • High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy

    Exclusion Criteria include:

    • Malignancy
    • Rheumatological/autoinflammatory/autoimmune disease
    • Metabolic disease
    • Leishmania infection

    Study Sites

    St. Jude Children’s Research Hospital, Memphis, Tennessee
    Collaborating sites outside the U.S.

  2. About this study

    Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition that affects the body’s immune system. The disorder most often is seen in babies and young children.  This study will collect information about HLH patients’ symptoms, laboratory values, treatment and outcomes.

    Purpose of this clinical trial

    The main goal of the study is to create an international database of HLH patient information. This information will help researchers develop clinical trials in the future on new HLH treatments.

    Eligibility overview

    • Diagnosis of a primary immunodeficiency predisposed to HLH
    • Meets at least 4 of the 8 diagnostic criteria for HLH and is receiving HLH-directed therapy
    • High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy
  3. HSESID Quick View
    Sponsors St. Jude Children’s Research Hospital    
    Trial Start Date May2017
    Estimated Enrollment 300 (30 at St. Jude)
    Study Type Observational
    Conditions Hemophagocytic lymphohistiocytosis
    Age All ages
    Principal investigator Melissa Hines, MD
    Study Sites St. Jude Children’s Research Hospital and collaborating sites in and outside the U.S.
    For a consultation or to discuss HSESID
    St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.